Hemophilia therapeutic gets orphan drug status

A drug therapy for hemophilia patients developed by CSL Behring has been given orphan drug status by U.S. regulators.

The hemophilia therapeutic from CSL Behring, a plasma protein therapeutics producer in King of Prussia, Pennsylvania, links recombinant fusion protein coagulation factor VIIa with albumin. The treatment is designed to help hemophilia A and hemophilia B patients with inhibitors.

Hemophilia is a bleeding disorder marked by prolonged bleeding, particularly into the muscles, joints or internal organs, caused by defective or deficient coagulation proteins, factor VIII.

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One complication associated with hemophilia is that patients develop inhibitor antibodies against normal factor VIII coagulation proteins because of frequent infusions. In hemophiliacs, the normal antibody is treated as foreign because the body does not have a copy, so factor VIII becomes ineffective.

Dr. Russell Basser, senior vice president of global clinical research and development at CSL Behring, said: “CSL Behring’s rVIIa albumin fusion protein is expected to exhibit a good tolerability profile and improved pharmacokinetics that may enable prophylaxis.”

Orphan status is given to treatments for rare diseases that affect less than 200,000 people in the U.S. It qualifies the company for tax credits, a waiver for U.S. Food and Drug Administration license application fees and gives certain marketing incentives.

The drug rVIIa-FP received orphan drug status from European regulators in May 2011.